Ascendis Pharma A/S (NASDAQ: ASND) will present two-year data from its ApproaCH trial of TransCon CNP at the International Skeletal Dysplasia Society (ISDS) annual meeting in Toronto this week.
The oral presentation, scheduled for Friday at 10:00 a.m. during Session 4, will cover Week 104 results from the open-label extension of the trial in children with achondroplasia. The data will be delivered by Carlos Bacino, M.D., Professor of Molecular and Human Genetics at Baylor College of Medicine and Texas Children's Hospital.
A separate poster presentation will analyze injection site reactions throughout the conference, addressing scenarios where indirect treatment comparisons are not feasible. TransCon CNP is a once-weekly prodrug of C-type natriuretic peptide designed to deliver continuous exposure of active CNP, counteracting overactive FGFR3 signaling associated with achondroplasia.
The company’s drug, branded as YUVIWEL, received U.S. FDA approval in February 2026 for increasing linear growth in pediatric patients aged two and older with achondroplasia and open epiphyses. In Europe, the Marketing Authorisation Application for YUVIWEL remains under review by the European Medicines Agency, with a decision expected in the fourth quarter of 2026.
Achondroplasia, a rare genetic disorder caused by a fibroblast growth factor receptor 3 variant, affects more than 250,000 people globally and is associated with complications including spinal abnormalities, enlarged brain ventricles, impaired muscle strength, hearing deficits, upper airway obstructions, and chronic pain.
